Patients Thought Untreatable with Rare Disease Dramatically Improve with Common Gene Therapy

A single-dose gene replacement therapy is found to transform the capabilities for movement in children over 2 years of age and teenagers with spinal muscular atrophy, according to research published in Nature Medicine. The effects allowed these minors who could sit but not stand to move like they’ve never done before, including standing up, walking, […] The post Patients Thought Untreatable with Rare Disease Dramatically Improve with Common Gene Therapy appeared first on Good News Network.